Gain-of-Function Research Is Vital to US Innovation

This brief is part of “Election 2024: Policy Playbook,” a series by Rice University and the Baker Institute that offers critical context, analysis, and recommendations to inform policymaking in the United States and Texas.

The Big Picture

• Gain-of-function research broadly refers to experiments that genetically alter an organism to give it new functions that can be used in agriculture, sustainability, and medicine.
• In a few cases, gain-of-function research increases the capability of an organism to cause disease with the purpose of understanding these mechanisms and developing effective therapies. 
• Existing research oversight ensures gain-of-function research is carefully reviewed prior to, during, and after funding.
• A widespread ban on gain-of-function research would impede innovation of tools, technologies and therapies that can influence wide-ranging areas of public concern such as medicine, energy, and sustainability.

Why It Matters

Gain-of-function research is an important scientific tool that can lead to major breakthroughs in developing ways to fight diseases such as cancer or to innovate in areas such as energy and sustainability. Enabled by synthetic biology, gain-of-function research involves the application of engineering principles to biology by genetically modifying organisms to give them new or enhanced abilities. 

Potential uses of synthetic biology include identifying emerging diseases; breaking down of toxic chemicals in soil and superfund sites (locations with high levels of toxins and other contaminants); developing more sustainable fertilizers to decrease agricultural costs; recycling waste into useful chemicals and materials; and creating new therapies for diseases such as cancer, diabetes, infectious diseases and other emerging threats to our health. This growing field has been designated as a future driver of the global economy, estimated to be worth $80 billion by 2033.

Summarizing the Debate

Debates surrounding gain-of-function research focus on biomedical scientific experiments that increase the ability of pathogens such as bacteria, viruses, and fungi to spread to humans or become more virulent. Manipulating and studying viruses is important because it helps researchers understand what makes them spread and what keeps them contained. However, in nature, cells are constantly sharing genetic material with other cells. Sometimes this results in new capabilities, the loss of former capabilities, or no change at all. It is critical to understand when these natural processes lead to beneficial innovations versus when they might pose threats to health, crops, or environment.

Gain-of-function data allows researchers to identify how a pathogen could potentially become a pandemic, strategize on how to prevent one, outwit a pathogen’s resistance to vaccines, and develop other therapies. But because researchers cannot always predict the outcome of their experiments, some gain-of-function studies may end up increasing pathogenicity instead of decreasing it. However, there are mechanisms in place that oversee this research to ensure the public is not harmed. But some policymakers may misunderstand how and why this research is conducted, leading to debates about the risks of conducting gain-of-function research.

Understanding the Controversy

Gain-of-function research gained notoriety during the COVID-19 pandemic, when public figures began to question whether the virus was a result of researchers manipulating a virus in a lab versus being transmitted from an animal. These debates led to discussions at state and federal levels regarding bans on gain-of-function research, often written with overly broad language. Because of this language, these bans would obstruct other areas of research in virology and synthetic biology, which work to maintain the United States’ competitive edge in the rapidly growing bioeconomy.

Oversight Mechanisms Already in Place

The language around these broad bans assumes there are no current regulatory mechanisms to ensure that any research conducted in the United States is performed safely with low risks of public exposure. In fact, there are several oversight mechanisms already in place, including policies for dual use research of concern. In 2017, the Department of Human Health Services established a specific framework on how to review and guide funding decisions for gain-of-function research. And when the COVID-19 pandemic reignited concerns on gain-of-function pathogen research, the debate that followed ultimately resulted in the National Science Advisory Board for Biosecurity’s recommendation for tighter restrictions. These guidelines were updated in 2024 and a new framework was developed to account for advances in DNA synthesis technology. Research institutions that receive federal funding (including all U.S. universities) must adhere to federal guidelines for genetic engineering or they risk losing funding entirely.

Overall, there are a variety of checks and balances already in place to protect researchers and the public.

The Consequences of a Ban

Policies to regulate or even ban gain-of-function research can have a wide-ranging influence beyond the potentially controversial work on viruses and pathogens. A ban or restrictive regulation can also impact all research in synthetic biology and its ability to develop novel functions in cells that can lead to new therapies and technologies benefiting a wide range of fields including medicine, agriculture, aquaculture, energy, pollution remediation, and even space exploration. Furthermore, a restriction or ban could negatively impact U.S. leadership in science, technology, and innovation — especially in the emerging global bioeconomy.

Policy Actions

Determining what qualifies as potentially dangerous gain-of-function research is crucial to improving regulatory structures that prioritize public safety and health without compromising on innovation or losing out on economic benefits. It is imperative that scientists and engineers work to define the boundaries of this research and develop strategies to communicate these boundaries to policymakers.

To avoid passing new legislation that could undermine public health, biosecurity, and the United States’ competitiveness in science, it is recommended that policymakers:

1. Develop a precise definition of synthetic biology and gain-of-function research to be used across the federal government.
2. Establish or empower both new and existing federal research oversight committees to review all gain-of-function research with a significant potential to increase pathogens.
3. Avoid passing new restrictions or regulations on research, especially those which broadly define gain-of-function research.

The Bottom Line

Gain-of-function research can be broadly defined to include large portions of virology, microbiology, molecular biology, synthetic biology, and other areas that are vital to transforming cells and viruses into new biotechnologies, therapies, and tools. New legislation is not needed; instead existing oversight mechanisms and their continuous updates will ensure that potentially dangerous research is adequately safeguarded.

This material may be quoted or reproduced without prior permission, provided appropriate credit is given to the author and Rice University’s Baker Institute for Public Policy. The views expressed herein are those of the individual author(s), and do not necessarily represent the views of Rice University’s Baker Institute for Public Policy.